NCT07125066View on ClinicalTrials.gov

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

PHASE1Enrolling by invitationINTERVENTIONAL

Enrollment

1

Participants

Timeline

Start Date

July 30, 2025

Primary Completion Date

July 31, 2027

Study Completion Date

December 31, 2027

Conditions

Combined D,L-2-hydroxyglutaric Aciduria

Interventions

DRUG

Sodium phenylbutyrate

open-label design with doses of sodium phenylbutyrate

Trial Locations (1)

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

Sponsors

Collaborators (1)

Zevra Therapeutics

All Listed Sponsors

collaborator

Zevra Therapeutics

INDUSTRY

lead

Jerry Vockley, MD, PhD

OTHER